Study of Fludarabine + Cyclophosphamide + TBI Conditioning Regimen for Double Units Cord Blood Transplantation(CBT)in Severe Aplastic Anemia(SAA)

Aplastic Anemia Clinical Trial

Official title:

Phase I/II Study of Fludarabine, Cyclophosphamide Plus TBI Conditioning Regimen for Double Units Cord Blood Transplantation in Severe Aplastic Anemia

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT00881933
• Other study ID #: KSPHO-SCT0804
• Status: Recruiting
• Phase: Phase 1/Phase 2
• First received: April 14, 2009
• Last updated: March 23, 2012
• Start date: November 2008
• Est. completion date: September 2012

Study information

• Verified date: March 2012
• Source: The Korean Society of Pediatric Hematology Oncology
• Contact: Hyoung Jin Kang, M.D, Ph.D
• Phone: 82 2 2072 3304
• Email: kanghj[@]snu.ac.kr
• Is FDA regulated: No
• Health authority: Korea: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

Severe aplastic anemia is a fatal disease and patients without HLA matched siblings need alternative treatment option. Cord blood transplantation (CBT) has become an alternative treatment means in various diseases, but it has not been proved to be good for severe aplastic anemia. Double units CBT is proposed to have better engraftment potential and and we reported successful double units UCBT after engraftment failure with single unit with promising result. To increase the engraftment potential, fludarabine, cyclophosphamide plus TBI conditioning regimen for double units cord blood transplantation was proposed for the patient with severe aplastic anemia without HLA-matched donor.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 5
• Est. completion date: September 2012
• Est. primary completion date: September 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 1 Year to 21 Years

Eligibility

Inclusion Criteria:

• Diagnosis of severe aplastic anemia defined by any two or three peripheral blood criteria and either marrow criterion.

• Peripheral blood

• Neutrophils < 0.5 x 109/l

• Platelets < 20 x 109/l

• Corrected reticulocytes < 1%

• Bone marrow

• Severe hypocellularity (< 25%)

• Moderate hypocellularity (25-30%) with hematopoietic cells representing < 30% of residual cells

• No prior hematopoietic stem cell transplantation.

• Age: no limits.

• Performance status: ECOG 0-2.

• Patients must be free of significant functional deficits in major organs, but the following eligibility criteria may be modified in individual cases.

• Heart: a shortening fraction > 30%, ejection fraction > 45%.

• Liver: total bilirubin < 2 × upper limit of normal; ALT < 3 × upper limit of normal.

• Kidney: creatinine <2 × normal or a creatinine clearance (GFR) > 60 ml/min/1.73m2.

• Patients must lack any active viral infections or active fungal infection.

• No appropriate donor

• Appropriate cord blood is available: matched at least in 4/6 of A, B, DR loci.

• Patients (or one of parents if patients age < 19) should sign informed consent.

Exclusion Criteria:

• Pregnant or nursing women.

• Malignant or nonmalignant illness that is uncontrolled or whose control may be jeopardized by complications of study therapy.

• Psychiatric disorder that would preclude compliance.

• Congenital aplastic anemia including Fanconi anemia.

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Aplastic Anemia

Intervention

Other:
• Cyclophosphosphamide, TBI
fludarabine (30 mg/m2 once daily i.v. on days -7, -6, -5, -4, -3, & -2) (if body weight < 10 kg or if age < 1 year: 1.33 mg/kg) cyclophosphamide (60 mg/kg once daily i.v. on days -9, & -8) total body irradiation (3G on days -1)

Locations

Country	Name	City	State
Korea, Republic of	Seoul National University Hospital	Seoul

Sponsors (1)

Lead Sponsor	Collaborator
The Korean Society of Pediatric Hematology Oncology

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To evaluate the engraftment potential of fludarabine, busulfan plus thymoglobulin conditioning regimen for cord blood transplantation (CBT) in severe aplastic anemia.		From Oct 2008. to Sep 2011	No
Secondary	To evaluate the incidence and severity of toxicity and treatment related mortality		From Oct 2008. to Sep 2011	No
Secondary	To evaluate overall and event free survival rate		From Oct 2008. to Sep 2011	No
Secondary	To evaluate acute and chronic GVHD		From Oct 2008. to Sep 2011	No
Secondary	To evaluate immunologic recovery after CBT		From Oct 2008. to Sep 2011	No